Status:
COMPLETED
Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
Lead Sponsor:
LYSOGENE
Conditions:
Mucopolysaccharidosis Type III A
Sanfilippo Disease Type A
Eligibility:
All Genders
18-6 years
Phase:
PHASE1
PHASE2
Brief Summary
The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vec...
Eligibility Criteria
Inclusion
- Age: 18 (eighteen) months to end of 6 (six) years
- Onset of clinical manifestations related to MPSIIIA during the first 5 years of life
- SGSH activity in peripheral blood cell and / or cultured fibroblast extracts of less than 10% of controls.
- Patient affiliated to the French social security or assimilated regimens
- Family understanding the procedure and the informed consent
- Signed informed consent
- Vital laboratory parameters within normal range
Exclusion
- Presence of brain atrophy on inclusion MRI judged on a cortico-dural distance of more than 1cm
- No independent walking (Ability to walk without help)
- Any condition that would contraindicate permanently anaesthesia
- Any other permanent medical condition not related to MPSIIIA
- Any vaccination 1 month before investigational drug administration
- Intake of aspirin within one month
- Any medication aiming at modifying the natural course of MPSIIIA given during the 6 months before vector injection
- Any condition that would contraindicate treatment with Prograf®, Modigraf®, Cellcept® and Solupred®
Key Trial Info
Start Date :
August 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2013
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT01474343
Start Date
August 1 2011
End Date
May 1 2013
Last Update
May 6 2014
Active Locations (2)
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1
Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris
Le Kremlin-Bicêtre, France, 94275
2
Hôpital Necker, Assistance Publique des Hôpitaux de Paris
Paris, France, 75015